A controlled clinical trial of ISIS 304801 in patients with Familial Chylomicronemia Syndrome (FCS)

Phase 1

• Conditions: Familial Chylomicronemia Syndrome (FCS); Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]; MedDRA version: 18.1Level: LLTClassification code 10017339Term: Fredrickson Type I lipidaemiaSystem Organ Class: 100000004850; MedDRA version: 18.1Level: LLTClassification code 10060593Term: Fredrickson Type I lipidemiaSystem Organ Class: 100000004850

• Registration Number: EUCTR2014-002421-35-GB
• Lead Sponsor: Ionis Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-07-23
• Last Update Posted: 22 May 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

• History of chylomicronemia
• A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
• Fasting triglycerides = 750 mg/dL (8.4 mmol/L) at Screening
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 70
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• Diabetes mellitus if newly diagnosed or if HbA1c = 9.0%
• Other types of severe hypertriglyceridemia
• Active pancreatitis within 4 weeks of screening
• Acute Coronary Syndrome or major surgery within 3 months of screening
• History of heart failure
• Treatment with Glybera gene therapy within 2 years of screening
• Previous treatment with APOCIII Rx
• Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified